April 17, 2025 RDCA-DAP Webinar: ACE Inhibitors as a Cardioprotective Treatment in Dystrophinopathies ACE inhibitors were the first pharmacological class shown to benefit heart failure treatment...
February 26, 2025 Transforming Data into Breakthroughs: 5 Years of RDCA-DAP Journey and Impact In honor of Rare Disease Month, Alexandre Bétourné took some time to sit down and discuss the impact RDCA-DAP has made...
February 24, 2025 Lysosomal Storage Disease Data Sharing Workshop, Webinar Series – Session #1: Setting the Scene C-Path's CPLD team presents, "Lysosomal Storage Disease Data Sharing Workshop, Webinar Series - Session #1...
February 20, 2025 Vivli and RDCA-DAP Present: Data Platform Partnerships to Promote Rare Disease Data Sharing Join Julie Wood, Chief Operating Officer at Vivli, for an engaging webinar, Thursday, February 20, where she’ll discuss...
January 21, 2025 Save the Date: CGIC Returns to D.C. this September We are thrilled to invite you to the 2025 Critical Path Institute® (C-Path) Global Impact Conference (CGIC)...
January 7, 2025 Inclusion in neurological research: empowering people living with neurological diseases The value of involving people living with diseases in the research process is increasingly recognized...
November 27, 2024 Autosomal Recessive Polycystic Kidney Disease (ARPKD)Adjunct EL-PFDD Scientific Workshop Report The mission of the Polycystic Kidney Disease Foundation is to fund research, advocate for patients, and ultimately to end PKD..
November 27, 2024 A computational tool to optimize clinical trial parameter selection in Duchenne muscular dystrophy: A practical guide and case studies Duchenne muscular dystrophy (DMD), a rare pediatric disease, presents numerous challenges when designing clinical trials...
December 1, 2024 Advancing rare disease measurement through the Rare Disease Clinical Outcome Assessment Consortium Naomi Knoble, and Lindsey T. Murray on behalf of the Rare Disease Clinical Outcome Assessment Consortium. View all authors and aff
September 30, 2024 TNF-α inhibitors for type 1 diabetes: exploring the path to a pivotal clinical trial Type 1 diabetes (T1D) is an autoimmune disease characterized by the destruction...
